Motor neurone disease breakthrough hope as drug that can ‘slow progression of ALS’ starts late stage trial

Thousands of people living with motor neurone disease could be given the chance to live longer, thanks to a new drug shown to slow the progression of the most common form of the degenerative disease.

Amyotrophic lateral sclerosis (ALS) is the most common form of motor neuron disease; Muscle wasting progressively damages some parts of the nervous system and cannot be treated.

It gradually prevents patients from moving, speaking, swallowing and even eating.

But scientists now believe they can slow the progression of the disease by preserving motor function and prolonging survival with a twice-daily pill called pridopidine.

Sigma-2 receptor (SIR) antagonist has previously been shown to play a role in stimulating multiple neuroprotective pathways implemented in neurodegenerative diseases such as ALS and Huntington’s Disease.

Clinical studies to date have demonstrated the safety and effectiveness of the drug, with data from more than 1,600 patients, some of whom have been on active treatment for seven years.

Yesterday, the drug’s manufacturers, Prilemia Therapeutics and Ferrer, announced their first participation in this important study on patients with rapidly progressing ALS.

Although not currently approved by any regulatory authority, researchers believe the global study, which will include more than 500 participants, could pave the way for therapeutic treatments that slow the progression of the disease.

Co-director of the Mass General Brigham Neurological Clinical Research Institute, Dr. Sabrina Paganoni said: ‘Enrolling the first participant in this confirmatory study is a milestone in our search for potential new treatment options that could help preserve function, sustain speech and prolong survival – the key goals of early ALS treatment.’

MND charities have welcomed the next phase of the trial, underlining the need for early diagnosis and treatment.

Kuldip Dave, Senior Vice President of Research at the ALS Association, said: ‘The ALS community urgently needs new treatment options that can delay the relentless progression of the disease and awaits the outcome of this study.

‘The earlier we can diagnose and treat ALS, the greater the potential to preserve function and quality of life for longer; These are the key to making ALS survivable until we can cure it.’

The PREVAiLS trial will be conducted at nearly 60 leading ALS treatment centers in 13 countries, including the US, EU and UK.

The 48-week placebo study is trying to enroll patients with definite or probable ALS who are within 18 months of the first appearance of their symptoms.

ALS caused the death of Eric Danes, the star of Grey’s Anatomy, at the beginning of this year at the age of 53, and the famous scientist Stephen Hawking also suffered from this disease.

Danes, who played the role of Dr. Mark Sloan, announced that he was diagnosed with ALS less than a year before his death.

The TV hunk is best known as Dr Mark Sloan, played by Patrick Dempsey in the 2006 series Grey’s Anatomy, alongside Dr Derek Shepherd, above.

It is not fully understood why MND occurs, and there is currently no treatment to halt the disease’s brutal course; Instead, doctors focus on relieving the worst of the symptoms.

Around 5,000 adults in the UK have the disease, putting them at a one in 300 risk of developing it in their lifetime.

The disease causes muscle weakness that gets worse over several months or years.

Symptoms typically first include stiff or weak hands, lesser weakness and feet that may cause the person to trip and fall a lot, and twitches, spasms, or muscle cramps.

As MND worsens, the patient may have problems breathing, swallowing, and speaking. Eventually they will no longer be able to walk or move.

About half of those with this condition have a life expectancy of only two to three years from the onset of symptoms. However, these can get worse quickly.

Some people can live up to 10 years, and in rarer cases even longer.